Manusiran (Divesiran) is a GalNac-siRNA targeting liver and transmembrane serine protease 6 (Serine protease 6). Manusiran increases hepatic Hepcidin synthesis and plasma levels by silencing TMPRSS6, a negative regulator of hepcidin production, and limits the availability of iron required for erythropoiesis. Combined use of Manusiran with Deferiprone (HY-B0568) reduces ineffective erythropoiesis and hepatic iron overload in a mouse model of beta-thalassemia. Manusiran can be used for research on polycythemia vera, type 1 hereditary hemochromatosis, and beta-thalassemia[1][2][3].
